RNA Based Gene Therapy In A Model Of RNA Based Gene Therapy In A Model Of Usher syndrome KU Leuven
Usher syndrome (USH) is the most prevalent cause of combined deafness and blindness in humans. USH is categorized in three distinct subtypes based on severity of hearing loss and presence of vestibular dysfunction. The hearing loss due to mutations in different USH genes results from disorganization of the hair bundles of the cochlear hair cells. In all three USH subtypes visual impairment is due to retinitis pigmentosa (RP). Mutations in ...